Reference-based sensitivity analysis for time-to-event data

被引:19
|
作者
Atkinson, Andrew [1 ,2 ]
Kenward, Michael G. [3 ]
Clayton, Tim [1 ]
Carpenter, James R. [1 ]
机构
[1] London Sch Hyg & Trop Med, Dept Med Stat, London, England
[2] Univ Bern, Univ Hosp Bern, Dept Infect Dis, Bern, Switzerland
[3] UCL, MRC Clin Trials Unit, London, England
基金
英国医学研究理事会;
关键词
missing data; MNAR; multiple imputation; sensitivity analysis; time to event; PROPORTIONAL HAZARDS ASSUMPTION; MULTIPLE IMPUTATION; ACCESSIBLE ASSUMPTIONS; CORONARY ANGIOPLASTY; LONGITUDINAL TRIALS; PROTOCOL DEVIATION; CLINICAL-TRIALS; MODEL; INFERENCE; FRAMEWORK;
D O I
10.1002/pst.1954
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
The analysis of time-to-event data typically makes the censoring at random assumption, ie, that-conditional on covariates in the model-the distribution of event times is the same, whether they are observed or unobserved (ie, right censored). When patients who remain in follow-up stay on their assigned treatment, then analysis under this assumption broadly addresses the de jure, or "while on treatment strategy" estimand. In such cases, we may well wish to explore the robustness of our inference to more pragmatic, de facto or "treatment policy strategy," assumptions about the behaviour of patients post-censoring. This is particularly the case when censoring occurs because patients change, or revert, to the usual (ie, reference) standard of care. Recent work has shown how such questions can be addressed for trials with continuous outcome data and longitudinal follow-up, using reference-based multiple imputation. For example, patients in the active arm may have their missing data imputed assuming they reverted to the control (ie, reference) intervention on withdrawal. Reference-based imputation has two advantages: (a) it avoids the user specifying numerous parameters describing the distribution of patients' postwithdrawal data and (b) it is, to a good approximation, information anchored, so that the proportion of information lost due to missing data under the primary analysis is held constant across the sensitivity analyses. In this article, we build on recent work in the survival context, proposing a class of reference-based assumptions appropriate for time-to-event data. We report a simulation study exploring the extent to which the multiple imputation estimator (using Rubin's variance formula) is information anchored in this setting and then illustrate the approach by reanalysing data from a randomized trial, which compared medical therapy with angioplasty for patients presenting with angina.
引用
收藏
页码:645 / 658
页数:14
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